Background: Despite international initiatives on collaboration within the field of rare
diseases, patient access to orphan medicinal products (OMPs) and healthcare services …

Background: Decision-makers have implemented a variety of value assessment frameworks
(VAFs) for orphan drugs in European jurisdictions, which has contributed to variations in …

Abstract Background and Objective Enthusiasm for the use of outcomes-based managed
entry agreements (OBMEAs) to manage uncertainties apparent at the time of …

Objectives Health technology assessment (HTA) uses evidence appraisal and synthesis
with economic evaluation to inform adoption decisions. Standard HTA processes sometimes …

Background The number of market approvals of orphan medicinal products (OMPs) has
been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for …

Objectives With complex health technologies entering the market, methods for health
technology assessment (HTA) may require changes. This study aimed to identify challenges …

Significant advances in understanding cardiac amyloidosis (CA) have been made in recent
years, leading to a thorough reformulation of its clinical significance. In addition to …

Health technology assessment (HTA) is increasingly informed by nonrandomized studies,
but there is limited guidance from HTA bodies on expectations around evidence quality and …

Background There is increasing recognition that conventional appraisal approaches may be
unsuitable for assessing the value rare disease treatments (RDTs). This research examines …

Rare diseases imply clinical and economic burden as well as a significant challenge for
health systems. One relevant objective of the activities planned within the European …